1. Is a Clinical Trial Right for Me?
Carol Burke, MD
Lisa Pedicone, PhD

2. What is a clinical trial?
A clinical trial is a research study
Clinical trials test whether a medical strategy, treatment or device is safe for humans and effective for disease diagnosis, prevention or treatment

3. What is a clinical trial?
The main purpose of a clinical trial is RESEARCH
To learn about risks and benefits of a medical intervention
Does a bile duct stent decrease my risk of bile duct obstruction
Does aspirin prevent heart attacks
Dose yoga improve back pain
Research studies usually compare two things
Standard of care vs a new approach
Placebo vs study agent
Patients must be aware that the research
May or may not benefit them
Could be associated with risks and harms

4. What are the steps needed to design a clinical trial?
Define a need for the research
Review currently available information on the topic
No information, information is controversial or scant
Develop a protocol
Background of disease or problem
Aims of the research
Outline what the treatment and anticipated outcomes will be
Identify the medical tests and visits for patients and frequency of evaluation
Plan to analyze results and determine if the trial was successful
Create inclusion and exclusion criteria to see who is eligible
Devise a patient safety and monitoring plan

5. What are the steps needed to design a clinical trial?
Create an informed consent document
Explains all facets of study to patients
Signed by research subject and investigator or designee
Design a budget
Approval of protocol, informed consent form, budget, monitoring plan from regulatory agencies

6. Members of the Study Team
Sponsor
Principal Investigator
Co Investigators
Research Coordinator
Institutional Review Board
Data Safety Monitoring Board
Other agencies (FDA, NCI, NIH)
Patient

7. Study Team Responsibilities
Being in a Research Trial is a BIG DEAL
Contractual Obligation
Abide by the protocol
Meeting, the visit schedule, taking your study agent
Keeping diaries of dosing, other medical issues/doctors visits, etc
Contacting research staff when they need you
Birth control, possible avoidance of other interventions
Benefits: There is no cost to you
In early phase studies, you may receive money to cover the costs of multiple office visits.
In most trials, the cost of the medication and all tests/procedures directly specified by the study are covered by the sponsoring company (typically pharmaceutical/device company).
You are followed closely, usually be experts in the field
You are contributing to science!

8. Types of Clinical Trials: Phases

9. Phase 1 First time drug is given to humans
First done in “healthy volunteers” and then in patients with the disease
Usually only given for 1-14 days
Not able to show if drug “works” but to show major safety signal
Small number of patients included
Typically involves more blood draws and physical exams than normal medical care

10. Phase 2 Studies patients with the disease
Usually treatment is given for a longer time to test whether the treatment works
Evaluates longer term safety
Typically very specific patient population (many exclusion criteria) and harder to qualify
Most times new treatment is tested against a control
Active control: Another drug known to have benefit in patients with the disease
Placebo control: A sham pill/injection
Most times patients will be ”randomized” to receive new treatment or the control (placebo or active)

11. Phase 3 Trials which are the focus of new drug approvals by FDA
Larger number of patients to test how well the treatment works and to measure adverse events
Some trials compare a new drug to an already approved drug to prove similar/better (active control trials)

12. Phase 4
Trials that test approved drugs (eg, vancomycin vs ursodiol) or procedures (eg, ERCP stenting vs balloon) in broader patient populations
Pediatric trials are usually performed after safety and efficacy are established in adults
Trials are typically conducted at a larger number of centers
Many times expanded to include more community/regional medical centers

13. Putting it all together…

14. The bad news…

15. How do I know the drug in the clinical trial is safe and works?
Simple Answer:
You don’t; however, what phase of development the trial is in can give you some information
More information on the drug known if drug is in later stage development

16. Do clinical trials only include drugs that haven’t been approved yet?
Simple Answer:
No, it depends on the phase the trial is in development
Phase 4 typically studies approved drugs or devices

17. How Do I Find a Clinical Trial?
Use the ”Clinical Trials” link on the PSC Partners page under tab Patients & Caregivers” (filters for PSC trials)
Talk to your doctor about possible trials in your area

18. What Do I Look For… What locations is the study being conducted at?
What are the inclusion and exclusion criteria? (very difficult to get a deviation approved)
How long will the study last?
What is likelihood I will receive the drug/treatment under study vs a control (how many “arms”)?
Is there a treatment arm or is objective non-treatment?
Blood/tissue samples for genetic research
Patient questionnaires relating to patient reported outcomes
Interventions such as diet modification

19. If I find a study that I qualify for, then what?
Carefully read the informed consent before signing
If a placebo controlled trial, ask if you will be offered the active drug after the study if you were randomized to placebo
Consider the time participating will require (travel time, extra visits beyond your normal care, extra tests, extra paperwork and questionnaires)
Can you handle the ’unknown’?
What if you don’t receive the new drug/treatment? Are you willing to remain in the study until the end?
Do you have other options?

20. Final words
Even if a study isn’t providing a new treatment, clinical research plays a very important part in working towards cures.
Registries (PSC Partners registry!) are critical sources of information especially for rare diseases.
Always consider the benefit:risk ratio when considering enrolling in any type of trial and decide what is best for your individual situation.

21. Questions